Base editing: advances and therapeutic opportunities

被引：264
|
作者：
Porto, Elizabeth M. [1 ,6 ]
Komor, Alexis C. [1 ,6 ]
Slaymaker, Ian M. [2 ]
Yeo, Gene W. [3 ,4 ,5 ]
机构：
[1] Univ Calif San Diego, Dept Chem & Biochem, La Jolla, CA 92093 USA
[2] Beam Therapeut, Synthet Biol Dept, Cambridge, MA USA
[3] Univ Calif San Diego, Dept Cellular & Mol Med, La Jolla, CA 92093 USA
[4] Univ Calif San Diego, Biomed Sci & Bioinformat Grad Program, La Jolla, CA 92093 USA
[5] Univ Calif San Diego, Syst Biol Grad Program, La Jolla, CA 92093 USA
[6] Univ Calif San Diego, Inst Genom Med, La Jolla, CA 92093 USA
来源：
NATURE REVIEWS DRUG DISCOVERY | 2020年 / 19卷 / 12期
基金：
美国国家卫生研究院;
关键词：
DUCHENNE MUSCULAR-DYSTROPHY; METABOLIC LIVER-DISEASE; MODIFIED MESSENGER-RNA; IN-VIVO; HIGHLY EFFICIENT; OFF-TARGET; GENE-EXPRESSION; CAENORHABDITIS-ELEGANS; NANOPARTICLE DELIVERY; CRISPR-CAS9; NUCLEASES;
D O I：
10.1038/s41573-020-0084-6
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
Base editing - the introduction of single-nucleotide variants (SNVs) into DNA or RNA in living cells - is one of the most recent advances in the field of genome editing. As around half of known pathogenic genetic variants are due to SNVs, base editing holds great potential for the treatment of numerous genetic diseases, through either temporary RNA or permanent DNA base alterations. Recent advances in the specificity, efficiency, precision and delivery of DNA and RNA base editors are revealing exciting therapeutic opportunities for these technologies. We expect the correction of single point mutations will be a major focus of future precision medicine.
引用
收藏
页码：839 / 859
页数：21
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